Real-World Evidence and the Food and Drug Administration

Based on applicable laws and regulations, the U.S. Food and Drug Administration (FDA) evaluates evidence submitted in support of applications for a new medical product or for a new indication involving an approved medical product. For decades, evidence from randomized controlled trials has been the basis for evaluating the therapeutic effectiveness and safety of drugs and biological products. More recently, however, electronic access to more detailed clinical data is evolving, and the reliability and relevance of such information in research are improving. The availability of more robust data on clinical factors that affect health outcomes has also provided opportunities for exploring various statistical methods in lieu of randomization. In parallel with these technical and methodological changes, the 21^st Century Cures Act of 2016 mandated that FDA develop a framework for evaluating the use of “real-world evidence” (RWE)—defined as “data regarding the usage, or the potential benefits or risks, of a drug derived from sources other than randomized clinical trials”—to support approvals of new indications for previously approved drugs and to support or fulfill post-approval study requirements. The corresponding term “real-world data” (RWD) refers to data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources. This presentation will focus on how the U.S. Food and Drug Administration (FDA) is adapting to shifting scientific paradigms and new legislation pertaining to the use of “RWE” in evaluating whether a drug works.